KDDF Global C&D Tech Fair

No.

106

Asset Title

Gene Therapy for Long-term Decrease of Intraocular Pressure

Organization

Duke University

Product Type

Gene therapy

Therapeutic Area

Ophthalmology

Development Stage

Target Identification or Validation

Technical Summary

• miR-146a was upregulated in senescent fibroblasts and trabecular meshwork (TM) cells and that its upregulation could act as a brake to excessive production of inflammatory cytokines that are part of the senescence-associated secretory phenotype.

• The TM is constantly subject to mechanical forces such as IOP spikes, cardiac cycle, blinking, and eye movement.

• miR-146 affected the expression of genes potentially involved in outflow homeostasis at basal levels and under CMS.

• Both lentiviral and adenoviral vectors expressing miR-146a resulted in sustained decreases in IOP ranging from 2.6 to 4.4 mmHg.

• Long term follow-up of rats injected with lentiviral vectors showed a sustained effect on IOP of 4.4 ± 2.9 mmHg that lasted until rats were sacrificed more than 8months later.

• Eyes showed no signs of inflammation, loss of visual acuity, or other visible abnormalities.

Patent

US20220273694A1

Publication

Long-Term Decrease of Intraocular Pressure in Rats by Viral Delivery of miR-146a. TVST (2021)
MicroRNA-24 Regulates the Processing of Latent TGFβ1 During Cyclic Mechanical Stress in Human Trabecular Meshwork Cells Through Direct Targeting of FURIN. J. Cell Physiol. (2011)
Modulation of inflammatory markers by miR-146a during replicative senescence in trabecular meshwork cells. IOVS (2010)

Attachment

Duke University_Overview_Gene Therapy for Long-term Decrease of Intraocular Pressure.pdf
Duke University_Pub_Long-Term Decrease of Intraocular Pressure in Rats by Viral Delivery of miR-146a.pdf
Duke University_Pub_MicroRNA-24 Regulates the Processing of Latent TGFβ1 During Cyclid Mechanical Stress.pdf
Duke University_Pub_Modulation of inflammatory markers by miR-146a during replicative senescence in trabecular meshwork cells.pdf